Azacitidine Is Effective in Patients with Low / Intermediate-1 Risk Myelodysplastic Syndrome Managed in Community Based Practice: Preliminary Data From the Spanish Compassionate Use Registry.

Myelodysplastic syndrome (MDS) is comprised of a group of heterogeneous hematological disorders. Although the decision regarding treatment of a patient with MDS is based on performance status, age, patient preference and concomitant illnesses, NCCN-recommended treatment approaches vary according to IPSS risk score. Azacitidine (AZA) is a hypomethylating agent recently approved in Europe for the treatment of MDS. AZA was available in Spain under compassionate use before its regulatory approval and marketing authorization from the Spanish Medicines Agency in May 2009.

We present the preliminary analysis of the clinical data from a longitudinal, multicenter Spanish patient registry. Data on the disease course and management of patients with MDS treated with AZA under compassionate use conditions were retrospectively collected from community-based hematology clinics. As of August 1, 2009, data from 90 patients MDS diagnosed according to WHO criteria had been collected with low / intermediate-1 International Prognostic Scoring System (IPSS) risk score.

At baseline, the median age was 70 years, the male/female ratio was 69/31, and the majority of patients had primary MDS (80 patients; 90%) and an ECOG performance status of 0-1 (62 patients; 69%). The most frequent initial dose of AZA administered was 75 mg/m² (77 patients, 86%). Almost all patients followed one of the following three schedules: days 1-7 (31%), days 1-5 and 8-9 (29%) or days 1-5 (37%) in a 28-day cycle. AZA was administered mostly subcutaneously (71 patients, 79%). The mean number of cycles administered was 7 (range 2-25). The overall treatment response was 61% (International Working Group 2006 criteria): 21% complete response, 11% complete bone marrow response, 3% partial response, 27% hematological response. In addition, 12% had stable disease. AZA was generally well tolerated. Grade 3/4 adverse events documented in these patients, regardless of their relationship to the active treatment, were neutropenia (48%), thrombocytopenia (36%), anemia (26%), febrile neutropenia (12%), rash (3%), injection site reaction (2%), asthenia (1%), septic shock (1%), constitutional symptoms (1%), and hypotension (1%).

These preliminary results demonstrate that, in a community-based setting, patients with low / intermediate-1 IPSS risk MDS respond to treatment with AZA, with an acceptable safety profile. More data derived from this registry and from the clinical trials are awaited.

No relevant conflicts of interest to declare.